FDA grants Retevmo accelerated approval for pediatric patients with thyroid cancer
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The FDA granted accelerated approval to selpercatinib for the treatment of certain pediatric patients with metastatic or advanced solid tumors harboring RET alterations.
The approval applies to children aged two years or older who require systemic therapy for advanced or metastatic medullary thyroid cancer with an RET mutation. The approval also applies to pediatric patients aged two years or older who require systemic therapy for radioactive iodine-refractory advanced or metastatic thyroid cancer with a RET gene fusion and those with locally advanced or metastatic solid tumors with a RET gene fusion who have experienced disease progression after previous systemic therapy with no alternative treatment options.
All three indications require confirmation of RET alterations via an FDA-approved test.
This is the first FDA approval of a targeted therapy for pediatric patients aged 12 years and younger with RET alterations. Selpercatinib received previous accelerated approval for thyroid cancer indications in adults and pediatric patients 12 years of age and older. The agent also received regular approval for adults with locally advanced or metastatic RET fusion-positive non-small cell lung cancer.
FDA approved the new indications based on results from the multicenter phase 1/phase 2 LIBRETTO-121 trial. The single-arm study evaluated selpercatinib 92 mg/m2 orally twice daily in 25 pediatric and young adult patients with locally advance or metastatic solid tumors harboring RET alterations that had not responded to available therapies or had no other available treatment options.
Study results showed an overall response rate of 48% (95% CI, 28-69) among patients treated with selpercatinib, with a median duration off response not yet reached.
Adverse reactions experienced by more than 25% of participants in the LIBRETTO-121 trial included musculoskeletal pain, diarrhea, headache, nausea, vomiting, coronavirus infection, abdominal pain, fatigue, pyrexia, and hemorrhage.
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